Community-engaged approaches to explore research priorities in Duchenne and Becker muscular dystrophy
Year: 2015
Type: dissertation
Study design: Observational Study
Abstract: This thesis presents a series of translational research studies to explore topics of importance to a patient stakeholder community--Duchenne and Becker muscular dystrophy. The overarching objective was to inform a patient/family foundation's interventions and policy and advocacy approaches.\n\nResults from a longitudinal study on mothers’ wellbeing support the need for systematic exploration of caregivers’ unmet support needs, especially those related to coping with DMD-related uncertainty and fear. Efforts to improve mothers’ adaptation should focus on fostering resilience and enhancing benefit finding.\n\nA study on treatment preferences and disease impact that used Best-Worst Scaling\nfound that caregivers were willing to accept a serious or fatal risk when balanced with a non-curative treatment, even absent lifespan improvement. Parents’ most pressing worries were about symptom progression and access to medical care.\n\nThe final qualitative studies highlight the complexity of clinical trial decision making, especially for rare, progressive pediatric disorders, where participants equated doing “nothing” with doing harm. Parents developed intentions to participate in trials before the informed consent process. Parents’ decisions were strongly influenced by the anticipation of individual benefit. The adaptive optimism engendered by the availability of trial was highly valued by both parents and clinicians on trial teams.
Author: Holly L. Peay
Language: English
Topic: Ethics in Clinical Research
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W2578659867